A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme
Study on Safety and Tolerability of Shorter Infusion for Investigational Medication Used in Treating Fabry Disease
Study Overview
This Phase 4 study will evaluate the safety and tolerability of Fabrazyme at current approved dose with increases in the infusion rate and reduced infusion volume. This study aims to generate data to provide the guidance on how infusion rate can be safely increased and minimize the burden of the life-long treatment with Fabrazyme.
Study Details
The total duration will be up to 7 months
Eligibility Criteria
You may be eligible for this study if you meet the following criteria:
- Conditions: Fabry's Disease
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Age: 2 years - 65 years
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Gender: All
Inclusion Criteria:
- Participants with confirmed diagnosis of FD who are ≥2 and ≤65 years of age at the time
of signing the informed consent form (ICF) or assent, if applicable.
- Cohort 1: female participants with body weight ≥30 kg who have been treated with Fabrazyme for at least 3 months without IARs during the most recent 3 infusions.
- Cohort 2: non-classic male participants with body weight ≥30 kg who have been treated with Fabrazyme for at least 3 months without IARs during the most recent 3 infusions.
- Cohort 3: classic male participants with body weight ≥30 kg who have been treated with Fabrazyme for at least 3 months without IARs during the most recent 3 infusions.
- Cohort 4: participants with body weight <30 kg who have been treated with Fabrazyme for at least 3 months without IARs during the most recent 3 infusions.
Women of childbearing potential must use a highly effective method of contraception through the study.
Exclusion Criteria:
- Female participants who are pregnant or breastfeeding.
- History of significant allergic disease or hypersensitivity to Fabrazyme or other medicinal products.
- Contraindication to Fabrazyme or any of the premedications or rescue medications (diphenhydramine, loratadine, cetirizine, fexofenadine, acetaminophen, montelukast, dexamethasone).
- Any other medical condition considered to make the increased infusion rate not tolerable at the Investigator's discretion.
The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.
This study investigates the safety and tolerability of changing the infusion rate and volume for an investigational medication used in treating Fabry disease. The purpose of this study is to explore how these changes might reduce the treatment burden for patients.
Participants will receive the investigational medication at a faster infusion rate and with a reduced volume. The study involves monitoring for any infusion-associated reactions (IARs) and other side effects to ensure safety. Participants will be grouped into different study arms based on their characteristics, such as gender and type of Fabry disease.
- Who can participate: Eligible participants are those aged 2 to 65 years with a confirmed diagnosis of Fabry disease. Participants must have been treated with the investigational medication for at least 3 months without infusion-associated reactions. Women of childbearing potential must use effective contraception.
- Study details: Participants will receive the investigational medication with adjustments in infusion rate and volume. They will be monitored for any infusion-associated reactions and other side effects. An inactive substance, known as a placebo, will not be used in this study.
- Study timelines: The study will last up to 7 months.
